A Phase 3 Study Of OCU400 Gene Therapy for the Treatment Of Retinitis Pigmentosa

NCTID	NCT06388200 (View at clinicaltrials.gov)
Description	This is a Phase 3 study to Assess the Efficacy, Safety and Tolerability of OCU400 in patients with retinitis pigmentosa (RP) associated with RHO mutations and patients with any other RP associated mutation with a clinical phenotype of RP. This is a multicenter, assessor blinded and randomized study which will enroll 150 subjects. (Show More)
Indication	Retinitis Pigmentosa
Compound Name	OCU400
Sponsor	Ocugen
Funder Type	Industry
Status	Recruiting
Enrollment Count	150

Target Gene/Variant	NR2E3
Therapy Type	Gene transfer
Therapy Route	In-vivo
Mechanism of Action	Overexpression of protective allele/gene
Route of Administration	Subretinal
Drug Product Type	Viral vector
Target Tissue/Cell
Delivery System	Viral transduction
Vector Type	AAV5
Editor Type
Dose 1	2.5 x 10^10 vg/eye
Dose 2
Dose 3
Dose 4
Dose 5

No.of Trial Sites	5
Locations	United States

Has US IND	True
Recent Updates	First patient dosed 6/20/24; on track for 2026 BLA and MAA approval targets